Research programme: autologous gene therapy - Orchard Therapeutics

Drug Profile

Research programme: autologous gene therapy - Orchard Therapeutics

Alternative Names: Ex-vivo lentiviral gene therapy - Orchard Therapeutics

Latest Information Update: 07 Dec 2016

Price : *
* Final gross price and currency may vary according to local VAT and billing address.

At a glance

  • Originator Orchard Therapeutics
  • Class Gene therapies
  • Mechanism of Action Gene transference
  • Orphan Drug Status

    Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

  • New Molecular Entity No

Highest Development Phases

  • Preclinical Immunological disorders; Metabolic disorders; Mucopolysaccharidosis III

Most Recent Events

  • 29 Nov 2016 Mucopolysaccharidosis-IIIA (MPS-IIIA or Sanfilippo Syndrome type A) and undisclosed follow-on indications
  • 21 Oct 2016 Orchard Therapeutics plans a first-in-man trial for Mucopolysaccharidosis III (Orchard Therapeutics website, October 2016)
  • 19 Oct 2016 Preclinical trials in Metabolic disorders in United Kingdom (Parenteral)
Restricted Access

Oops, it looks like you don’t have a valid subscription to this content. To gain full access to the content and functionality of the AdisInsight database try one of the following.

  • with a username/password associated to an account with a valid subscription
  • Contact your organization’s admin about adding this content to your AdisInsight subscription
  • Request a trial

If you are a subscriber to this content then contact us at so we can help.

Back to top